Gaucher disease and SARS-CoV-2 infection: Emerging management challenges.
Identifieur interne : 000561 ( Main/Exploration ); précédent : 000560; suivant : 000562Gaucher disease and SARS-CoV-2 infection: Emerging management challenges.
Auteurs : Pramod Mistry [États-Unis] ; Manisha Balwani [États-Unis] ; Deborah Barbouth [États-Unis] ; T Andrew Burrow [États-Unis] ; Edward I. Ginns [États-Unis] ; Ozlem Goker-Alpan [États-Unis] ; Gregory A. Grabowski [États-Unis] ; Reena V. Kartha [États-Unis] ; Priya S. Kishnani [États-Unis] ; Heather Lau [États-Unis] ; Chung U. Lee [États-Unis] ; Grisel Lopez [États-Unis] ; Gustavo Maegawa [États-Unis] ; Seymour Packman [États-Unis] ; Carlos Prada [États-Unis] ; Barry Rosenbloom [États-Unis] ; Tamanna Roshan Lal [États-Unis] ; Rapheal Schiffmann [États-Unis] ; Neal Weinreb [États-Unis] ; Ellen Sidransky [États-Unis]Source :
- Molecular genetics and metabolism [ 1096-7206 ] ; 2020.
Descripteurs français
- KwdFr :
- Appréciation des risques (MeSH), Betacoronavirus (MeSH), Humains (MeSH), Infections à coronavirus (complications), Infections à coronavirus (épidémiologie), Maladie de Gaucher (complications), Maladie de Gaucher (thérapie), Maladies transmissibles émergentes (thérapie), Maladies transmissibles émergentes (épidémiologie), Pandémies (MeSH), Pneumopathie virale (complications), Pneumopathie virale (épidémiologie), Prévision (MeSH).
- MESH :
- thérapie : Maladie de Gaucher, Maladies transmissibles émergentes.
- épidémiologie : Infections à coronavirus, Maladies transmissibles émergentes, Pneumopathie virale.
- complications : Appréciation des risques, Betacoronavirus, Humains, Infections à coronavirus, Maladie de Gaucher, Pandémies, Pneumopathie virale, Prévision.
English descriptors
- KwdEn :
- Betacoronavirus (MeSH), COVID-19 (MeSH), Communicable Diseases, Emerging (epidemiology), Communicable Diseases, Emerging (therapy), Coronavirus Infections (complications), Coronavirus Infections (epidemiology), Forecasting (MeSH), Gaucher Disease (complications), Gaucher Disease (therapy), Humans (MeSH), Pandemics (MeSH), Pneumonia, Viral (complications), Pneumonia, Viral (epidemiology), Risk Assessment (MeSH), SARS-CoV-2 (MeSH).
- MESH :
- complications : Coronavirus Infections, Gaucher Disease, Pneumonia, Viral.
- epidemiology : Communicable Diseases, Emerging, Coronavirus Infections, Pneumonia, Viral.
- therapy : Communicable Diseases, Emerging, Gaucher Disease.
- Betacoronavirus, COVID-19, Forecasting, Humans, Pandemics, Risk Assessment, SARS-CoV-2.
DOI: 10.1016/j.ymgme.2020.05.002
PubMed: 32471800
PubMed Central: PMC7211677
Affiliations:
- États-Unis
- Arkansas, Californie, Caroline du Nord, Connecticut, District de Columbia, Floride, Maryland, Massachusetts, Minnesota, Ohio, Texas, Virginie, État de New York
- Washington (district de Columbia)
Links toward previous steps (curation, corpus...)
Le document en format XML
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<country xml:lang="fr">États-Unis</country>
<wicri:regionArea>Departments of Pediatrics, and Molecular Genetics and Biochemistry, University of Cincinnati College of Medicine, Cincinnati Children's Hospital Medical Center, Cincinnati, OH</wicri:regionArea>
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<author><name sortKey="Kartha, Reena V" sort="Kartha, Reena V" uniqKey="Kartha R" first="Reena V" last="Kartha">Reena V. Kartha</name>
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<author><name sortKey="Kishnani, Priya S" sort="Kishnani, Priya S" uniqKey="Kishnani P" first="Priya S" last="Kishnani">Priya S. Kishnani</name>
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<author><name sortKey="Maegawa, Gustavo" sort="Maegawa, Gustavo" uniqKey="Maegawa G" first="Gustavo" last="Maegawa">Gustavo Maegawa</name>
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<author><name sortKey="Prada, Carlos" sort="Prada, Carlos" uniqKey="Prada C" first="Carlos" last="Prada">Carlos Prada</name>
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<country xml:lang="fr">États-Unis</country>
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<country xml:lang="fr">États-Unis</country>
<wicri:regionArea>Medical Genetics Branch, National Human Genome Research Institute, NIH, Bethesda, MD</wicri:regionArea>
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<sourceDesc><biblStruct><analytic><title xml:lang="en">Gaucher disease and SARS-CoV-2 infection: Emerging management challenges.</title>
<author><name sortKey="Mistry, Pramod" sort="Mistry, Pramod" uniqKey="Mistry P" first="Pramod" last="Mistry">Pramod Mistry</name>
<affiliation wicri:level="2"><nlm:affiliation>Department of Internal Medicine and Pediatrics,Yale School of Medicine, New Haven, CT, United States of America. Electronic address: pramod.mistry@yale.edu.</nlm:affiliation>
<country xml:lang="fr">États-Unis</country>
<wicri:regionArea>Department of Internal Medicine and Pediatrics,Yale School of Medicine, New Haven, CT</wicri:regionArea>
<placeName><region type="state">Connecticut</region>
</placeName>
</affiliation>
</author>
<author><name sortKey="Balwani, Manisha" sort="Balwani, Manisha" uniqKey="Balwani M" first="Manisha" last="Balwani">Manisha Balwani</name>
<affiliation wicri:level="2"><nlm:affiliation>Department of Genetics and Genomic Sciences, Icahn School of Medicine at Mount Sinai, NY, United States of America. Electronic address: manisha.balwani@mssm.edu.</nlm:affiliation>
<country xml:lang="fr">États-Unis</country>
<wicri:regionArea>Department of Genetics and Genomic Sciences, Icahn School of Medicine at Mount Sinai, NY</wicri:regionArea>
<placeName><region type="state">État de New York</region>
</placeName>
</affiliation>
</author>
<author><name sortKey="Barbouth, Deborah" sort="Barbouth, Deborah" uniqKey="Barbouth D" first="Deborah" last="Barbouth">Deborah Barbouth</name>
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<country xml:lang="fr">États-Unis</country>
<wicri:regionArea>Department of Human Genetics, University of Miami, Miller School of Medicine</wicri:regionArea>
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<author><name sortKey="Burrow, T Andrew" sort="Burrow, T Andrew" uniqKey="Burrow T" first="T Andrew" last="Burrow">T Andrew Burrow</name>
<affiliation wicri:level="2"><nlm:affiliation>Department of Human Genetics, University of Miami, Miller School of Medicine, United States of America; Department of Pediatrics, University of Arkansas for Medical Sciences and Arkansas Children's Hospital, Section of Genetics and Metabolism, Little Rock, AR, United States of America. Electronic address: TABurrow@uams.edu.</nlm:affiliation>
<country xml:lang="fr">États-Unis</country>
<wicri:regionArea>Department of Human Genetics, University of Miami, Miller School of Medicine, United States of America; Department of Pediatrics, University of Arkansas for Medical Sciences and Arkansas Children's Hospital, Section of Genetics and Metabolism, Little Rock, AR</wicri:regionArea>
<placeName><region type="state">Arkansas</region>
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<author><name sortKey="Ginns, Edward I" sort="Ginns, Edward I" uniqKey="Ginns E" first="Edward I" last="Ginns">Edward I. Ginns</name>
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<country xml:lang="fr">États-Unis</country>
<wicri:regionArea>Lysosomal Disorders Treatment and Research Program, Departments of Psychiatry and Neurology, University of Massachusetts Medical School, Worcester, MA</wicri:regionArea>
<placeName><region type="state">Massachusetts</region>
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<author><name sortKey="Goker Alpan, Ozlem" sort="Goker Alpan, Ozlem" uniqKey="Goker Alpan O" first="Ozlem" last="Goker-Alpan">Ozlem Goker-Alpan</name>
<affiliation wicri:level="2"><nlm:affiliation>Lysosomal and Rare Disorders Research and Treatment Center, Fairfax, VA, United States of America. Electronic address: ogoker-alpan@ldrtc.org.</nlm:affiliation>
<country xml:lang="fr">États-Unis</country>
<wicri:regionArea>Lysosomal and Rare Disorders Research and Treatment Center, Fairfax, VA</wicri:regionArea>
<placeName><region type="state">Virginie</region>
</placeName>
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<author><name sortKey="Grabowski, Gregory A" sort="Grabowski, Gregory A" uniqKey="Grabowski G" first="Gregory A" last="Grabowski">Gregory A. Grabowski</name>
<affiliation wicri:level="2"><nlm:affiliation>Departments of Pediatrics, and Molecular Genetics and Biochemistry, University of Cincinnati College of Medicine, Cincinnati Children's Hospital Medical Center, Cincinnati, OH, United States of America. Electronic address: Grabgo317@comcast.net.</nlm:affiliation>
<country xml:lang="fr">États-Unis</country>
<wicri:regionArea>Departments of Pediatrics, and Molecular Genetics and Biochemistry, University of Cincinnati College of Medicine, Cincinnati Children's Hospital Medical Center, Cincinnati, OH</wicri:regionArea>
<placeName><region type="state">Ohio</region>
</placeName>
</affiliation>
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<author><name sortKey="Kartha, Reena V" sort="Kartha, Reena V" uniqKey="Kartha R" first="Reena V" last="Kartha">Reena V. Kartha</name>
<affiliation wicri:level="2"><nlm:affiliation>Center for Orphan Drug Research, Department of Experimental and Clinical Pharmacology, University of Minnesota, Minneapolis, MN, United States of America. Electronic address: rvkartha@umn.edu.</nlm:affiliation>
<country xml:lang="fr">États-Unis</country>
<wicri:regionArea>Center for Orphan Drug Research, Department of Experimental and Clinical Pharmacology, University of Minnesota, Minneapolis, MN</wicri:regionArea>
<placeName><region type="state">Minnesota</region>
</placeName>
</affiliation>
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<author><name sortKey="Kishnani, Priya S" sort="Kishnani, Priya S" uniqKey="Kishnani P" first="Priya S" last="Kishnani">Priya S. Kishnani</name>
<affiliation wicri:level="2"><nlm:affiliation>Division of Medical Genetics, Department of Pediatrics, Duke University Medical Center, Durham, NC, United States of America. Electronic address: priya.kishnani@duke.edu.</nlm:affiliation>
<country xml:lang="fr">États-Unis</country>
<wicri:regionArea>Division of Medical Genetics, Department of Pediatrics, Duke University Medical Center, Durham, NC</wicri:regionArea>
<placeName><region type="state">Caroline du Nord</region>
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<author><name sortKey="Lau, Heather" sort="Lau, Heather" uniqKey="Lau H" first="Heather" last="Lau">Heather Lau</name>
<affiliation wicri:level="2"><nlm:affiliation>Division of Neurogenetics, Department of Neurology, New York University, New York, NY, United States of America. Electronic address: Heather.Lau@nyulangone.org.</nlm:affiliation>
<country xml:lang="fr">États-Unis</country>
<wicri:regionArea>Division of Neurogenetics, Department of Neurology, New York University, New York, NY</wicri:regionArea>
<placeName><region type="state">État de New York</region>
</placeName>
</affiliation>
</author>
<author><name sortKey="Lee, Chung U" sort="Lee, Chung U" uniqKey="Lee C" first="Chung U" last="Lee">Chung U. Lee</name>
<affiliation wicri:level="2"><nlm:affiliation>Lucile Packard Children's Hospital Stanford, Department of Pediatrics, Division of Medical Genetics, Stanford University School of Medicine, Palo Alta, CA, United States of America. Electronic address: chunglee@stanford.edu.</nlm:affiliation>
<country xml:lang="fr">États-Unis</country>
<wicri:regionArea>Lucile Packard Children's Hospital Stanford, Department of Pediatrics, Division of Medical Genetics, Stanford University School of Medicine, Palo Alta, CA</wicri:regionArea>
<placeName><region type="state">Californie</region>
</placeName>
</affiliation>
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<author><name sortKey="Lopez, Grisel" sort="Lopez, Grisel" uniqKey="Lopez G" first="Grisel" last="Lopez">Grisel Lopez</name>
<affiliation wicri:level="2"><nlm:affiliation>Medical Genetics Branch, National Human Genome Research Institute, NIH, Bethesda, MD, United States of America. Electronic address: glopez@mail.nih.gov.</nlm:affiliation>
<country xml:lang="fr">États-Unis</country>
<wicri:regionArea>Medical Genetics Branch, National Human Genome Research Institute, NIH, Bethesda, MD</wicri:regionArea>
<placeName><region type="state">Maryland</region>
</placeName>
</affiliation>
</author>
<author><name sortKey="Maegawa, Gustavo" sort="Maegawa, Gustavo" uniqKey="Maegawa G" first="Gustavo" last="Maegawa">Gustavo Maegawa</name>
<affiliation wicri:level="2"><nlm:affiliation>Division of Genetics and Metabolism, Departments of Pediatrics, Neuroscience, Molecular Genetics and Microbiology, University of Florida, Gainesville, FL, United States of America. Electronic address: gmaegawa@ufl.edu.</nlm:affiliation>
<country xml:lang="fr">États-Unis</country>
<wicri:regionArea>Division of Genetics and Metabolism, Departments of Pediatrics, Neuroscience, Molecular Genetics and Microbiology, University of Florida, Gainesville, FL</wicri:regionArea>
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</placeName>
</affiliation>
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<author><name sortKey="Packman, Seymour" sort="Packman, Seymour" uniqKey="Packman S" first="Seymour" last="Packman">Seymour Packman</name>
<affiliation wicri:level="2"><nlm:affiliation>Department of Pediatrics, Division of Medical Genetics, University of California San Francisco, San Francisco, CA, United States of America. Electronic address: Seymour.Packman@ucsf.edu.</nlm:affiliation>
<country xml:lang="fr">États-Unis</country>
<wicri:regionArea>Department of Pediatrics, Division of Medical Genetics, University of California San Francisco, San Francisco, CA</wicri:regionArea>
<placeName><region type="state">Californie</region>
</placeName>
</affiliation>
</author>
<author><name sortKey="Prada, Carlos" sort="Prada, Carlos" uniqKey="Prada C" first="Carlos" last="Prada">Carlos Prada</name>
<affiliation wicri:level="2"><nlm:affiliation>Division of Human Genetics, Cincinnati Children's Hospital Medical Center, Department of Pediatrics, University of Cincinnati College of Medicine, Cincinnati, OH, United States of America. Electronic address: carlos.prada@cchmc.org.</nlm:affiliation>
<country xml:lang="fr">États-Unis</country>
<wicri:regionArea>Division of Human Genetics, Cincinnati Children's Hospital Medical Center, Department of Pediatrics, University of Cincinnati College of Medicine, Cincinnati, OH</wicri:regionArea>
<placeName><region type="state">Ohio</region>
</placeName>
</affiliation>
</author>
<author><name sortKey="Rosenbloom, Barry" sort="Rosenbloom, Barry" uniqKey="Rosenbloom B" first="Barry" last="Rosenbloom">Barry Rosenbloom</name>
<affiliation wicri:level="2"><nlm:affiliation>Cedars-Sinai Tower Hematology Oncology, Beverly Hills, CA, United States of America. Electronic address: Barry.Rosenbloom@cshs.org.</nlm:affiliation>
<country xml:lang="fr">États-Unis</country>
<wicri:regionArea>Cedars-Sinai Tower Hematology Oncology, Beverly Hills, CA</wicri:regionArea>
<placeName><region type="state">Californie</region>
</placeName>
</affiliation>
</author>
<author><name sortKey="Lal, Tamanna Roshan" sort="Lal, Tamanna Roshan" uniqKey="Lal T" first="Tamanna Roshan" last="Lal">Tamanna Roshan Lal</name>
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<country xml:lang="fr">États-Unis</country>
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<region type="state">District de Columbia</region>
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<affiliation wicri:level="2"><nlm:affiliation>Baylor, Scott & White Research Institute, Dallas, TX, United States of America. Electronic address: Raphael.Schiffmann@bswhealth.org.</nlm:affiliation>
<country xml:lang="fr">États-Unis</country>
<wicri:regionArea>Baylor, Scott & White Research Institute, Dallas, TX</wicri:regionArea>
<placeName><region type="state">Texas</region>
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<author><name sortKey="Weinreb, Neal" sort="Weinreb, Neal" uniqKey="Weinreb N" first="Neal" last="Weinreb">Neal Weinreb</name>
<affiliation wicri:level="2"><nlm:affiliation>Departments of Human Genetics and Internal Medicine, University of Miami Miller School of Medicine, Miami, FL, United States of America. Electronic address: nweinreb@med.miami.edu.</nlm:affiliation>
<country xml:lang="fr">États-Unis</country>
<wicri:regionArea>Departments of Human Genetics and Internal Medicine, University of Miami Miller School of Medicine, Miami, FL</wicri:regionArea>
<placeName><region type="state">Floride</region>
</placeName>
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</author>
<author><name sortKey="Sidransky, Ellen" sort="Sidransky, Ellen" uniqKey="Sidransky E" first="Ellen" last="Sidransky">Ellen Sidransky</name>
<affiliation wicri:level="2"><nlm:affiliation>Medical Genetics Branch, National Human Genome Research Institute, NIH, Bethesda, MD, United States of America. Electronic address: sidranse@mail.nih.gov.</nlm:affiliation>
<country xml:lang="fr">États-Unis</country>
<wicri:regionArea>Medical Genetics Branch, National Human Genome Research Institute, NIH, Bethesda, MD</wicri:regionArea>
<placeName><region type="state">Maryland</region>
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<series><title level="j">Molecular genetics and metabolism</title>
<idno type="eISSN">1096-7206</idno>
<imprint><date when="2020" type="published">2020</date>
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<term>COVID-19 (MeSH)</term>
<term>Communicable Diseases, Emerging (epidemiology)</term>
<term>Communicable Diseases, Emerging (therapy)</term>
<term>Coronavirus Infections (complications)</term>
<term>Coronavirus Infections (epidemiology)</term>
<term>Forecasting (MeSH)</term>
<term>Gaucher Disease (complications)</term>
<term>Gaucher Disease (therapy)</term>
<term>Humans (MeSH)</term>
<term>Pandemics (MeSH)</term>
<term>Pneumonia, Viral (complications)</term>
<term>Pneumonia, Viral (epidemiology)</term>
<term>Risk Assessment (MeSH)</term>
<term>SARS-CoV-2 (MeSH)</term>
</keywords>
<keywords scheme="KwdFr" xml:lang="fr"><term>Appréciation des risques (MeSH)</term>
<term>Betacoronavirus (MeSH)</term>
<term>Humains (MeSH)</term>
<term>Infections à coronavirus (complications)</term>
<term>Infections à coronavirus (épidémiologie)</term>
<term>Maladie de Gaucher (complications)</term>
<term>Maladie de Gaucher (thérapie)</term>
<term>Maladies transmissibles émergentes (thérapie)</term>
<term>Maladies transmissibles émergentes (épidémiologie)</term>
<term>Pandémies (MeSH)</term>
<term>Pneumopathie virale (complications)</term>
<term>Pneumopathie virale (épidémiologie)</term>
<term>Prévision (MeSH)</term>
</keywords>
<keywords scheme="MESH" qualifier="complications" xml:lang="en"><term>Coronavirus Infections</term>
<term>Gaucher Disease</term>
<term>Pneumonia, Viral</term>
</keywords>
<keywords scheme="MESH" qualifier="epidemiology" xml:lang="en"><term>Communicable Diseases, Emerging</term>
<term>Coronavirus Infections</term>
<term>Pneumonia, Viral</term>
</keywords>
<keywords scheme="MESH" qualifier="therapy" xml:lang="en"><term>Communicable Diseases, Emerging</term>
<term>Gaucher Disease</term>
</keywords>
<keywords scheme="MESH" qualifier="thérapie" xml:lang="fr"><term>Maladie de Gaucher</term>
<term>Maladies transmissibles émergentes</term>
</keywords>
<keywords scheme="MESH" qualifier="épidémiologie" xml:lang="fr"><term>Infections à coronavirus</term>
<term>Maladies transmissibles émergentes</term>
<term>Pneumopathie virale</term>
</keywords>
<keywords scheme="MESH" xml:lang="en"><term>Betacoronavirus</term>
<term>COVID-19</term>
<term>Forecasting</term>
<term>Humans</term>
<term>Pandemics</term>
<term>Risk Assessment</term>
<term>SARS-CoV-2</term>
</keywords>
<keywords scheme="MESH" qualifier="complications" xml:lang="fr"><term>Appréciation des risques</term>
<term>Betacoronavirus</term>
<term>Humains</term>
<term>Infections à coronavirus</term>
<term>Maladie de Gaucher</term>
<term>Pandémies</term>
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<Month>06</Month>
<Day>22</Day>
</DateCompleted>
<DateRevised><Year>2021</Year>
<Month>01</Month>
<Day>10</Day>
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<JournalIssue CitedMedium="Internet"><Volume>130</Volume>
<Issue>3</Issue>
<PubDate><Year>2020</Year>
<Month>07</Month>
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<Title>Molecular genetics and metabolism</Title>
<ISOAbbreviation>Mol Genet Metab</ISOAbbreviation>
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<ArticleTitle>Gaucher disease and SARS-CoV-2 infection: Emerging management challenges.</ArticleTitle>
<Pagination><MedlinePgn>164-169</MedlinePgn>
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<Initials>P</Initials>
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<ForeName>Manisha</ForeName>
<Initials>M</Initials>
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<ForeName>Deborah</ForeName>
<Initials>D</Initials>
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</AffiliationInfo>
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<ForeName>T Andrew</ForeName>
<Initials>TA</Initials>
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<ForeName>Ozlem</ForeName>
<Initials>O</Initials>
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<ForeName>Gregory A</ForeName>
<Initials>GA</Initials>
<AffiliationInfo><Affiliation>Departments of Pediatrics, and Molecular Genetics and Biochemistry, University of Cincinnati College of Medicine, Cincinnati Children's Hospital Medical Center, Cincinnati, OH, United States of America. Electronic address: Grabgo317@comcast.net.</Affiliation>
</AffiliationInfo>
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<Author ValidYN="Y"><LastName>Kartha</LastName>
<ForeName>Reena V</ForeName>
<Initials>RV</Initials>
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<ForeName>Priya S</ForeName>
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</AffiliationInfo>
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<Author ValidYN="Y"><LastName>Lau</LastName>
<ForeName>Heather</ForeName>
<Initials>H</Initials>
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</AffiliationInfo>
</Author>
<Author ValidYN="Y"><LastName>Lee</LastName>
<ForeName>Chung U</ForeName>
<Initials>CU</Initials>
<AffiliationInfo><Affiliation>Lucile Packard Children's Hospital Stanford, Department of Pediatrics, Division of Medical Genetics, Stanford University School of Medicine, Palo Alta, CA, United States of America. Electronic address: chunglee@stanford.edu.</Affiliation>
</AffiliationInfo>
</Author>
<Author ValidYN="Y"><LastName>Lopez</LastName>
<ForeName>Grisel</ForeName>
<Initials>G</Initials>
<AffiliationInfo><Affiliation>Medical Genetics Branch, National Human Genome Research Institute, NIH, Bethesda, MD, United States of America. Electronic address: glopez@mail.nih.gov.</Affiliation>
</AffiliationInfo>
</Author>
<Author ValidYN="Y"><LastName>Maegawa</LastName>
<ForeName>Gustavo</ForeName>
<Initials>G</Initials>
<AffiliationInfo><Affiliation>Division of Genetics and Metabolism, Departments of Pediatrics, Neuroscience, Molecular Genetics and Microbiology, University of Florida, Gainesville, FL, United States of America. Electronic address: gmaegawa@ufl.edu.</Affiliation>
</AffiliationInfo>
</Author>
<Author ValidYN="Y"><LastName>Packman</LastName>
<ForeName>Seymour</ForeName>
<Initials>S</Initials>
<AffiliationInfo><Affiliation>Department of Pediatrics, Division of Medical Genetics, University of California San Francisco, San Francisco, CA, United States of America. Electronic address: Seymour.Packman@ucsf.edu.</Affiliation>
</AffiliationInfo>
</Author>
<Author ValidYN="Y"><LastName>Prada</LastName>
<ForeName>Carlos</ForeName>
<Initials>C</Initials>
<AffiliationInfo><Affiliation>Division of Human Genetics, Cincinnati Children's Hospital Medical Center, Department of Pediatrics, University of Cincinnati College of Medicine, Cincinnati, OH, United States of America. Electronic address: carlos.prada@cchmc.org.</Affiliation>
</AffiliationInfo>
</Author>
<Author ValidYN="Y"><LastName>Rosenbloom</LastName>
<ForeName>Barry</ForeName>
<Initials>B</Initials>
<AffiliationInfo><Affiliation>Cedars-Sinai Tower Hematology Oncology, Beverly Hills, CA, United States of America. Electronic address: Barry.Rosenbloom@cshs.org.</Affiliation>
</AffiliationInfo>
</Author>
<Author ValidYN="Y"><LastName>Lal</LastName>
<ForeName>Tamanna Roshan</ForeName>
<Initials>TR</Initials>
<AffiliationInfo><Affiliation>Rare Disease Institute, Children's National Medical Center, Washington DC, United States of America. Electronic address: troshanlal@childrensnational.org.</Affiliation>
</AffiliationInfo>
</Author>
<Author ValidYN="Y"><LastName>Schiffmann</LastName>
<ForeName>Rapheal</ForeName>
<Initials>R</Initials>
<AffiliationInfo><Affiliation>Baylor, Scott & White Research Institute, Dallas, TX, United States of America. Electronic address: Raphael.Schiffmann@bswhealth.org.</Affiliation>
</AffiliationInfo>
</Author>
<Author ValidYN="Y"><LastName>Weinreb</LastName>
<ForeName>Neal</ForeName>
<Initials>N</Initials>
<AffiliationInfo><Affiliation>Departments of Human Genetics and Internal Medicine, University of Miami Miller School of Medicine, Miami, FL, United States of America. Electronic address: nweinreb@med.miami.edu.</Affiliation>
</AffiliationInfo>
</Author>
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<ForeName>Ellen</ForeName>
<Initials>E</Initials>
<AffiliationInfo><Affiliation>Medical Genetics Branch, National Human Genome Research Institute, NIH, Bethesda, MD, United States of America. Electronic address: sidranse@mail.nih.gov.</Affiliation>
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<Month>05</Month>
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<QualifierName UI="Q000628" MajorTopicYN="Y">therapy</QualifierName>
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<region><li>Arkansas</li>
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